Healthcare Engineering Research Group, Centre for Biological Engineering, Wolfson School of Mechanical and Manufacturing Engineering, Loughborough University, Loughborough, Leicestershire LE11 3TU, UK.
Br Med Bull. 2013;108:73-94. doi: 10.1093/bmb/ldt034. Epub 2013 Nov 7.
Apart from haematopoietic stem cell transplantation for haematological disorders many stem cell-based therapies are experimental. However, with only 12 years between human embryonic stem cell isolation and the first clinical trial, development of stem cell products for regenerative medicine has been rapid and numerous clinical trials have begun to investigate their therapeutic potential.
This review summarizes key clinical trial data, current and future perspectives on stem cell-based products undergoing clinical trials, based on literature search and author research.
It is widely recognized that the ability to stimulate stem cell differentiation into specialized cells for use as cellular therapies will revolutionize health care and offer major hope for numerous diseases for which there are limited or no therapeutic options.
Stem cell-based products are unique and cover a large range of disorders to be treated; therefore, there is significant potential for variation in cell source, type, processing manipulation, the bioprocessing approach and scalability, the cost and purity of manufacture, final product quality and mode of action. As such there are gaps in regulatory and manufacturing frameworks and technologies, only a small number of products are currently within late phase clinical trials and few products have achieved commercialization.
Recent developments are encouraging acceleration through the difficulties encountered en route to clinical trials and commercialization of stem cell therapies.
The field is growing year on year with the first clinical trial using induced pluripotent stem cells anticipated by end 2013.
除了针对血液系统疾病的造血干细胞移植，许多基于干细胞的疗法都还处于试验阶段。然而，自从人类胚胎干细胞分离到首例临床试验仅仅过去了 12 年，再生医学中干细胞产品的开发已经十分迅速，并且已经开始了许多临床试验来探究它们的治疗潜力。
本综述基于文献检索和作者研究，总结了正在进行临床试验的基于干细胞的产品的关键临床试验数据、当前和未来的观点。
人们普遍认为，刺激干细胞分化为特定细胞以用于细胞疗法的能力将彻底改变医疗保健，并为许多治疗方法有限或没有的疾病带来重大希望。
基于干细胞的产品是独特的，涵盖了广泛的待治疗疾病；因此，细胞来源、类型、处理操作、生物处理方法和可扩展性、制造的成本和纯度、最终产品质量和作用模式都有很大的变化潜力。因此，监管和制造框架和技术存在差距，目前只有少数产品处于后期临床试验阶段，并且很少有产品实现商业化。
最近的发展令人鼓舞，有望克服临床试验和干细胞疗法商业化过程中遇到的困难。
该领域逐年发展，预计到 2013 年底将进行首例使用诱导多能干细胞的临床试验。
